Title

Exact two-stage designs for phase II activity trials with rank-based endpoints

Document Type

Article

Publication Date

3-2012

Publication Title

Contemporary Clinical Trials

Volume

33

Issue

2

First page number:

332

Last page number:

341

Abstract

Features common to phase II clinical trials include limited knowledge of the experimental treatment being evaluated, design components reflecting ethical considerations, and small to moderate sample sizes as a result of resource constraints. It is for these reasons that there exist many two-stage designs proposed in the literature for use in this context. The majority of these designs are for binary endpoints and based on exact probability calculations, or are for continuous endpoints and rooted in asymptotic approximations to the null distribution. We present exact two-stage Mann–Whitney designs in the context of two-arm randomized clinical trials. In addition to describing the designs, we present tables of decision rules under a variety of assumed realities for use in trial planning.

Keywords

Clinical trials; Exact test; Mann–Whitney test; Minimax design; Nonparametric; Nonparametric statistics; Optimal design; Phase II design; Structural optimization

Disciplines

Medicine and Health Sciences | Statistical Methodology | Statistics and Probability | Vital and Health Statistics

Language

English

Permissions

Use Find in Your Library, contact the author, or interlibrary loan to garner a copy of the item. Publisher policy does not allow archiving the final published version. If a post-print (author's peer-reviewed manuscript) is allowed and available, or publisher policy changes, the item will be deposited.

Identifier

DOI: http://dx.doi.org/10.1016/j.cct.2011.10.008

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